NEW YORK, NY and PETACH TIKVAH, ISRAEL--(Marketwired - April 28, 2014) - BrainStorm Cell Therapeutics (OTCQB: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today that the US Food and Drug Administration (FDA) has approved commencement of its Phase II clinical trial with NurOwn™ in patients with Amyotrophic Lateral Sclerosis (ALS). The trial will be launched initially at the Massachusetts General Hospital (MGH) in Boston, MA and the University of Massachusetts Memorial (UMass) Hospital in Worcester, MA following Institutional Review Board (IRB) approvals. Dana-Farber Cancer Institute's Connell O'Reilly Cell Manipulation Core Facility will manufacture the NurOwn™ cells for these two clinical sites. The trial will also be conducted at the Mayo Clinic in Rochester, Minnesota.
"Today's announcement represents the most significant milestone BrainStorm has achieved to date," said Mr. Chaim Lebovits, President. "More importantly, in our view, it has positive implications for the entire ALS community, indicating that the FDA recognizes the significant clinical potential of transplantation with our autologous, differentiated mesenchymal stem cells. We are excited to begin the US trial and are optimistic that the Phase II data will confirm the positive indications of clinical benefit we observed in earlier studies. This trial will be the first Phase II double-blinded stem cell study to be conducted for ALS."
"Following successful completion of the technology transfer process for BrainStorm's NurOwn cells, we are looking forward to collaborating on this multi-center study as the cell production facility for the Massachusetts clinical sites," added Professor Jerome Ritz, Department of Medicine, Harvard Medical School, and Director, Connell O'Reilly Cell Manipulation and Gene Transfer Laboratory, Dana-Farber Cancer Institute.
BrainStorm's Phase II trial is a randomized, double-blind, placebo controlled multi-center study designed to evaluate the safety and efficacy of transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors ("MSC-NTF" or NurOwn™) in 48 ALS patients. The NurOwn™ cells will be administered via combined intramuscular and intrathecal injection. Patients will be followed monthly for approximately three months before transplantation and six months following transplantation. The primary objective of the study will be safety and tolerability, as defined by a measure of the number of patients with adverse events. Secondary endpoints will measure a change in the Amyotrophic Lateral Sclerosis (ALS) Functional Rating Scale (ALS-FRS) and a change in Slow Vital Capacity (SVC) slopes. For further details, please go to http://1.usa.gov/1l5XWkW.
Earlier clinical trials have shown that treatment with NurOwn™ cells was well tolerated and safe. According to the final clinical study report for BrainStorm's Phase I trial in Israel, the study successfully met its primary and secondary endpoints. Moreover, there were "encouraging signals of efficacy in individuals receiving NurOwn treatment, in which a dramatic improvement was observed during more than two consecutive visits following treatment in one or more of their secondary efficacy endpoints (ALSFRS-R score, total neurological examination score, FVC, and CMAP). Initial observations of the company's Phase IIa trial data in Israel appear to reinforce these findings. An interim update is expected to be released later this quarter.
NurOwn is an autologous, adult stem cell therapy technology that differentiates bone marrow-derived mesenchymal stem cells (MSC) into "MSC-NTF" cells. These neuron-supporting cells secrete elevated levels of neurotrophic, or nerve-growth, factors for PROTECTION of existing motor neurons, PROMOTION of motor neuron growth, and RE-ESTABLISHMENT of nerve-muscle interaction. The ability to differentiate mesenchymal stem cells into MSC-NTF cells, and confirmation of their activity and potency before transplantation, makes NurOwn a first-of-its-kind approach for treating neurodegenerative diseases. More information about NurOwn™ can be found at http://brainstorm-cell.com/index.php/science-a-technology/-nurown.
About BrainStorm Cell Therapeutics, Inc.
BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. For more information, visit the company's website at www.brainstorm-cell.com.
Safe Harbor Statement
Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.